Florida Drug Discovery’s Big Need

Kevin Nash, Ph.D., an assistant professor of molecular pharmacology and physiology at the University of South Florida’s Byrd Alzheimer’s Institute, is on a mission against disease. Nash studies a small protein called Fractalkine, which essentially can quiet the immune system, and he’s searching for drugs that can achieve the same results in patients with Alzheimer’s and Parkinson’s.

Kyle Rohde, Ph.D. , assistant professor of infectious disease at the University of Central Florida’s College of Medicine, Burnett School of Biomedical Sciences, has similar designs on his target, tuberculosis. Rohde aims to discover novel chemical compounds capable of killing the bacterium Mycobacterium tuberculosis (Mtb) under conditions that mimic the in vivo environment during infection.

Nash and Rohde are two researchers among scores of others in university laboratories across Florida that share passion, knowledge — and the need for help.

In not so scientific terms, they can only go so far on their own.

“I don’t have the expertise to do this myself. So, this program enables me to look for molecules or compounds that I couldn’t otherwise do,” says Nash.

Engine Running Low

Sanford-Burnham Medical Research Institute at Lake Nona

“This program” is the Florida Translational Research Program, FTRP for short, established in 2011 by Gov. Rick Scott, and led by scientists at Sanford Burnham Prebys Medical Discovery Institute in Lake Nona. Five years ago, the idea was to rev the state’s economic engine with an emphasis on drug discovery, leveraging the technologies and talent existing within Lake Nona’s rising medical city outside of Orlando as a catalyst. These days, while the results can be measured in patents and commercial interest in the discoveries that have been made to date, funding of the drug discovery program is tenuous. The state has successfully created a pipeline of potential medicines by giving Florida-based research scientists access to SBP’s advanced drug discovery technology platform and expertise. Yet, such an effective funding stream was shut off in the 2015-16 state budget.

Nash’s project stalled last year when FTRP funding was cut off by the state. In an effort to find funds to bootstrap the project, he and the SBP team have since applied for funding from the National Institutes of Health (NIH) and the Alzheimer’s Drug Discovery Foundation (ADDF), although neither typically funds the earliest stage of drug discovery research.

“I cannot screen for any other compounds,” Nash continues. “Their expertise [at SBP] enables us to further the work that we’re doing. So, with FTRP support we’ll find compounds that will activate and we can further collaborate with Sanford Burnham Prebys.”

This spring, with the Florida Legislature still in session, that goal hangs in the balance.

Kyle Rohde, Ph.D.

For Rohde, the plan was to work through the FTRP to lay the foundation for new antibiotics to treat TB. His project was accepted in 2014 for early-stage work on assay development. Any further progress is now on hold.

“I can’t do what they [SBP scientists] do; there’s no way I could ever do that. They’re unique in their instrumentation and their expertise in executing a type of pretty intense, rigorous assays,” he comments.

“The type of money it would take to get these compounds and the things that we get through this collaboration with SBP, it’s prohibitive for any kind of new scientist who doesn’t already have a grant. You’re caught with ‘how do I get the data for the grant if I need the money from the grant to do that extensive research?’”

In addition, Rohde cites, disruptions to statewide collaborations have occurred, or have the potential to occur. He partners with USF in Tampa, Florida International University in Miami, the Harbor Branch Oceanographic Institution in Fort Pierce (a part of Florida Atlantic University) and the nearby Torrey Pines Institute for Molecular Studies in Port St. Lucie.

Collaboration Bridges Gap

FTRP’s place on the drug discovery landscape — in the “valley of death” — makes it especially vital. That valley is the gap between the laboratory bench and clinical phase testing. On average, an estimated $2.6 billion over 15 years is required to take a drug from discovery to commercialization.

Upon acceptance of proposals, FTRP gives basic researchers at Florida nonprofit institutes and universities, like Nash and Rohde, unprecedented opportunities to translate lab discoveries into new treatments. They are working together to conquer disease, with each FTRP project devoted to either a specific disease, such as cancer or broader research area like rare diseases.

“This program provides resources for high throughput screening and also establishes collaborations for multiple institutes and centers to enhance our research efforts,” says Xin (Cindy) Qi, Ph.D., an assistant professor in the Department of Medicinal Chemistry, College of Pharmacy, at the University of Florida.

Beginning in 2014, Qi worked with SBP researchers on developing novel therapeutics for narcolepsy and additional exploration of neurological pathways related to this disease. “It’s important to establish synergetic efforts within these programs across the state. More of these efforts will benefit everyone,” she adds.

Shaun Brothers, Ph.D.

For Shaun Brothers, Ph.D., associate director of the Center for Therapeutic Innovation and an assistant professor in the Department of Psychiatry and Behavioral Sciences, University of Miami Miller School of Medicine, “everyone” can be taken almost literal. Trained as a drug discovery pharmacologist, he focuses on the identification and preclinical development of new small molecules that have the potential to become therapies to treat cancer.

Three of his projects, submitted or co-submitted with associates, have been accepted by FTRP. On one of them, major funding from the National Institutes of Health is being sought in collaboration with SBP, among others. On another project, a global expert has tested FTRP-discovered compounds and confirmed them as highly promising potential cancer treatments that act in a new way relative to older drugs.

“Collaborating with SBP,” says Brothers, “we have identified three different sets of molecules that have the potential to be developed into tools to search for new targets to treat cancer.”

Indeed, big wins for everyone — including the state of Florida.

Concludes Rohde: “This is a way to uniquely make Florida researchers more competitive to bring in more federal dollars, which is more jobs and all sorts of good stuff for the state.”

About the Author

Candelaria is a 30-year veteran of the magazine industry in Central Florida, having served as a writer and editor for numerous publications. Those publications range from city/regional and business magazines to travel/leisure and sports magazines. Formerly, he was the editor of Orlando magazine and the 1996 president of the Florida Magazine Association. He is well-known in the Central Florida community, working with high-profile organizations such as the Orlando Regional Chamber of Commerce, Metro Orlando Economic Development Commission and Visit Orlando. He is a graduate of the University of Central Florida.